Modalis Therapeutics Unveils Epigenome Editing Therapy MDL-101 for Treatment of LAMA2-Deficient CMD
Shots:
- Modalis Therapeutics presented supportive preclinical data of MDL-101 in the preprinted paper “Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo” on bioRxiv, to treat LAMA2-CMD
- In the preclinical study, a single dose of MDL-101 using MYOAAV (an AAV vector) elevated LAMA1 in skeletal muscles for 12 mos., improved muscle pathophysiology, prolonged lifespan, and increased body weight of dyW mouse
- In adult & juvenile NHPs, MYOAAV version MDL-101 has shown enhanced muscle tropism and induced LAMA1 to function restoration level across skeletal muscle tissues without AEs, depicting its clinical efficacy
Ref: Modalis Therapeutics | Image: Modalis Therapeutics
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
